AU2014284152B2 - Compositions and methods for modulation of target nucleic acids - Google Patents

Compositions and methods for modulation of target nucleic acids Download PDF

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Publication number
AU2014284152B2
AU2014284152B2 AU2014284152A AU2014284152A AU2014284152B2 AU 2014284152 B2 AU2014284152 B2 AU 2014284152B2 AU 2014284152 A AU2014284152 A AU 2014284152A AU 2014284152 A AU2014284152 A AU 2014284152A AU 2014284152 B2 AU2014284152 B2 AU 2014284152B2
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substituted
certain embodiments
compound
alkyl
nucleoside
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AU2014284152A1 (en
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Stanley T. Crooke
Garth A. Kinberger
Walter F. Lima
Heather MURRAY
Thazha P. Prakash
Eric E. Swayze
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Ionis Pharmaceuticals Inc
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Ionis Pharmaceuticals Inc
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    • C—CHEMISTRY; METALLURGY
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    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/70—Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088—Compounds having three or more nucleosides or nucleotides
    • A61K31/7125—Nucleic acids or oligonucleotides having modified internucleoside linkage, i.e. other than 3'-5' phosphodiesters
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K47/00—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
    • A61K47/50—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates
    • A61K47/51—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent
    • A61K47/54—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent the modifying agent being an organic compound
    • A61K47/549—Sugars, nucleosides, nucleotides or nucleic acids
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P3/00—Drugs for disorders of the metabolism
    • A61P3/06—Antihyperlipidemics
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P43/00—Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/10—Type of nucleic acid
    • C12N2310/11—Antisense
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/10—Type of nucleic acid
    • C12N2310/14—Type of nucleic acid interfering nucleic acids [NA]
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/31—Chemical structure of the backbone
    • C12N2310/312—Phosphonates
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/32—Chemical structure of the sugar
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/33—Chemical structure of the base
    • C12N2310/334—Modified C
    • C12N2310/3341—5-Methylcytosine
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/34—Spatial arrangement of the modifications
    • C12N2310/346—Spatial arrangement of the modifications having a combination of backbone and sugar modifications
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/35—Nature of the modification
    • C12N2310/351—Conjugate
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/35—Nature of the modification
    • C12N2310/351—Conjugate
    • C12N2310/3515—Lipophilic moiety, e.g. cholesterol
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    • C12N2320/00—Applications; Uses
    • C12N2320/50—Methods for regulating/modulating their activity
    • C12N2320/51—Methods for regulating/modulating their activity modulating the chemical stability, e.g. nuclease-resistance
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    • C12N2320/00—Applications; Uses
    • C12N2320/50—Methods for regulating/modulating their activity
    • C12N2320/53—Methods for regulating/modulating their activity reducing unwanted side-effects

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  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Saccharide Compounds (AREA)
  • Measuring Or Testing Involving Enzymes Or Micro-Organisms (AREA)
AU2014284152A 2013-06-21 2014-06-23 Compositions and methods for modulation of target nucleic acids Active AU2014284152B2 (en)

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US201361838190P 2013-06-21 2013-06-21
US61/838,190 2013-06-21
US201361871683P 2013-08-29 2013-08-29
US61/871,683 2013-08-29
PCT/US2014/043731 WO2014205451A2 (en) 2013-06-21 2014-06-23 Compositions and methods for modulation of target nucleic acids

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EP (3) EP3011028B1 (enExample)
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AU (2) AU2014284152B2 (enExample)
CA (1) CA2916252A1 (enExample)
WO (1) WO2014205451A2 (enExample)

Families Citing this family (74)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
BRPI0923225A2 (pt) 2008-12-02 2016-10-04 Chiralgen Ltd metodo para sintese de acidos nucleicos modificados no atomo de fosforo
EP2451461A4 (en) 2009-07-06 2013-05-29 Ontorii Inc NOVEL NUCLEIC ACID PRODRUGS AND METHOD OF USE THEREOF
DK2620428T3 (da) 2010-09-24 2019-07-01 Wave Life Sciences Ltd Asymmetrisk hjælpegruppe
CN107365339A (zh) 2011-07-19 2017-11-21 波涛生命科学有限公司 合成官能化核酸的方法
WO2014010250A1 (en) 2012-07-13 2014-01-16 Chiralgen, Ltd. Asymmetric auxiliary group
PL2992098T3 (pl) 2013-05-01 2019-09-30 Ionis Pharmaceuticals, Inc. Kompozycje i sposoby modulowania ekspresji hbv i ttr
WO2015006740A2 (en) * 2013-07-11 2015-01-15 Alnylam Pharmaceuticals, Inc. Oligonucleotide-ligand conjugates and process for their preparation
EP3094728B1 (en) 2014-01-16 2022-03-09 Wave Life Sciences Ltd. Chiral design
WO2015168618A2 (en) 2014-05-01 2015-11-05 Isis Pharmaceuticals, Inc. Compositions and methods for modulating growth hormone receptor expression
CR20160554A (es) * 2014-05-01 2017-03-07 Ionis Pharmaceuticals Inc Composiciones y métodos para modular la expresión del factor b del complemento
US10570169B2 (en) 2014-05-22 2020-02-25 Ionis Pharmaceuticals, Inc. Conjugated antisense compounds and their use
EP3151839A4 (en) * 2014-06-06 2018-02-28 Ionis Pharmaceuticals, Inc. Compositions and methods for enhanced intestinal absorption of conjugated oligomeric compounds
AU2015329974B2 (en) * 2014-10-10 2019-06-20 F. Hoffmann-La Roche Ag GaINAc phosphoramidites, nucleic acid conjugates thereof and their use
AU2016332900C1 (en) 2015-09-29 2024-07-04 Amgen Inc. ASGR inhibitors
AU2016349625B2 (en) 2015-11-06 2022-07-07 Ionis Pharmaceuticals, Inc. Modulating apolipoprotein (a) expression
LT4119569T (lt) * 2015-11-06 2024-09-25 Ionis Pharmaceuticals, Inc. Konjuguoti priešprasmiai junginiai, skirti panaudoti terapijoje
TWI859114B (zh) 2016-04-11 2024-10-21 加拿大商艾爾布圖斯生技公司 靶向組合物
UY37376A (es) 2016-08-26 2018-03-23 Amgen Inc Construcciones de arni para inhibir expresión de asgr1 y métodos para su uso
WO2018067900A1 (en) * 2016-10-06 2018-04-12 Ionis Pharmaceuticals, Inc. Method of conjugating oligomeric compounds
US20180193270A1 (en) 2016-11-29 2018-07-12 PureTech Health LLC Exosomes for delivery of therapeutic agents
CN108239644B (zh) * 2016-12-23 2021-05-28 苏州瑞博生物技术股份有限公司 一种小干扰核酸和药物组合物及其用途
CA3059426A1 (en) 2017-04-11 2018-10-18 Arbutus Biopharma Corporation Targeted compositions
SG11201912178VA (en) * 2017-09-11 2020-01-30 Arrowhead Pharmaceuticals Inc Rnai agents and compositions for inhibiting expression of apolipoprotein c-iii (apoc3)
SG11202002149XA (en) 2017-09-14 2020-04-29 Janssen Biopharma Inc Galnac derivatives
WO2019105414A1 (zh) 2017-12-01 2019-06-06 苏州瑞博生物技术有限公司 一种核酸、含有该核酸的组合物与缀合物及制备方法和用途
CN118291456A (zh) 2017-12-01 2024-07-05 苏州瑞博生物技术股份有限公司 一种核酸、含有该核酸的组合物与缀合物及制备方法和用途
HRP20250277T1 (hr) 2017-12-01 2025-05-09 Suzhou Ribo Life Science Co., Ltd. Dvolančani oligonukleotid, sastav i konjugat koji sadrži dvolančani oligonukleotid, postupak njihove pripreme i upotreba
JP7360716B2 (ja) 2017-12-01 2023-10-13 スーチョウ リボ ライフ サイエンス カンパニー、リミテッド 核酸、当該核酸を含む組成物および複合体ならびに調製方法と使用
CN110944675B9 (zh) 2017-12-01 2024-08-09 苏州瑞博生物技术股份有限公司 一种核酸、含有该核酸的组合物与缀合物及制备方法和用途
AR113490A1 (es) 2017-12-12 2020-05-06 Amgen Inc CONSTRUCCIONES DE ARNi PARA INHIBIR LA EXPRESIÓN DE PNPLA3 Y MÉTODOS DE USO DE LAS MISMAS
WO2020038377A1 (zh) 2018-08-21 2020-02-27 苏州瑞博生物技术有限公司 一种核酸、含有该核酸的药物组合物和缀合物及其用途
US12049620B2 (en) 2018-09-11 2024-07-30 Amgen Inc. Purification methods for guanine-rich oligonucleotides
JP7376952B2 (ja) * 2018-09-30 2023-11-09 スーチョウ リボ ライフ サイエンス カンパニー、リミテッド ｓｉＲＮＡ複合体及びその調製方法と使用
AU2019380628B2 (en) * 2018-11-13 2024-10-31 Silence Therapeutics Gmbh Nucleic acids for inhibiting expression of LPA in a cell
GB2579253A (en) * 2018-11-28 2020-06-17 Pedanius Therapeutics Ltd Antibacterial antisense agents
CA3121510A1 (en) 2018-12-10 2020-06-18 Amgen Inc. Rnai constructs for inhibiting pnpla3 expression and methods of use thereof
SG11202105900UA (en) 2018-12-10 2021-07-29 Amgen Inc CHEMICALLY-MODIFIED RNAi CONSTRUCTS AND USES THEREOF
JP7507495B2 (ja) 2018-12-28 2024-06-28 スーチョウ リボ ライフ サイエンス カンパニー、リミテッド 核酸、当該核酸を含む組成物及び複合体ならびに調製方法と使用
CN113227376B (zh) 2019-05-22 2024-04-09 苏州瑞博生物技术股份有限公司 核酸、药物组合物与缀合物及制备方法和用途
JP7610848B2 (ja) 2019-05-22 2025-01-09 スーチョウ リボ ライフ サイエンス カンパニー、リミテッド 核酸、薬物組成物及び複合体ならびに調製方法と使用
CN113286888B (zh) 2019-05-22 2023-12-22 苏州瑞博生物技术股份有限公司 核酸、药物组合物与缀合物及制备方法和用途
CN113795582B (zh) 2019-05-24 2024-05-24 苏州瑞博生物技术股份有限公司 核酸、药物组合物与缀合物及制备方法和用途
SG11202113112WA (en) 2019-05-30 2021-12-30 Amgen Inc Rnai constructs for inhibiting scap expression and methods of use thereof
CA3143047A1 (en) 2019-06-25 2020-12-30 Amgen Inc. Purification methods for carbohydrate-linked oligonucleotides
US20220340911A1 (en) 2019-08-13 2022-10-27 Amgen Inc. Rnai constructs for inhibiting slc30a8 expression and methods of use thereof
KR20220062517A (ko) * 2019-08-15 2022-05-17 아이오니스 파마수티컬즈, 인코포레이티드 결합 변형된 올리고머 화합물 및 이의 용도
IL290766B2 (en) * 2019-09-03 2026-03-01 Arcturus Therapeutics Inc Administration of therapeutically active conjugates mediated by asialoglycoprotein receptor
WO2021054370A1 (ja) * 2019-09-18 2021-03-25 国立大学法人東京医科歯科大学 核酸複合体
JP7827623B2 (ja) 2019-12-09 2026-03-10 アムジエン・インコーポレーテツド ＬＰＡ発現を阻害するためのＲＮＡｉコンストラクト及び方法
CN115397989A (zh) 2020-02-18 2022-11-25 阿尔尼拉姆医药品有限公司 载脂蛋白C3(APOC3)iRNA组合物及其使用方法
EP4126967A1 (en) 2020-03-23 2023-02-08 Amgen Inc. Monoclonal antibodies to chemically-modified nucleic acids and uses thereof
US20230279399A1 (en) 2020-06-01 2023-09-07 Amgen Inc. Rnai constructs for inhibiting hsd17b13 expression and methods of use thereof
EP4196584A2 (en) 2020-08-13 2023-06-21 Amgen Inc. Rnai constructs and methods for inhibiting marc1 expression
US20240092819A1 (en) * 2020-10-20 2024-03-21 Sanofi Novel ligands for asialoglycoprotein receptor
KR20230104200A (ko) 2020-11-05 2023-07-07 암젠 인크 LPA-표적화된 RNAi 작제물을 이용하여 죽상경화성 심혈관질환을 치료하는 방법
EP4175964A1 (en) * 2021-01-30 2023-05-10 E-Therapeutics plc Conjugated oligonucleotide compounds, methods of making and uses thereof
CN117355534B (zh) 2021-01-30 2026-05-01 唐格拉姆治疗公司 缀合寡核苷酸化合物、其制备方法和用途
WO2022162161A1 (en) * 2021-01-30 2022-08-04 E-Therapeutics Plc Conjugated oligonucleotide compounds, methods of making and uses thereof
CN115850358B (zh) * 2021-03-08 2026-04-24 南京吉迈生物技术有限公司 GalNAc簇亚磷酰胺和靶向治疗性核苷
WO2022246195A1 (en) * 2021-05-20 2022-11-24 Olix Us, Inc. Functional moieties and their uses and synthetic preparation
CN117580953B (zh) * 2021-07-16 2025-07-18 苏州瑞博生物技术股份有限公司 一种核酸、含有该核酸的组合物与缀合物及制备方法和用途
CN118019566A (zh) 2021-09-30 2024-05-10 美国安进公司 分离富含鸟嘌呤的寡核苷酸的分子物质的方法
US20250011779A1 (en) 2021-10-05 2025-01-09 Amgen Inc. Compositions and methods for enhancing gene silencing activity of oligonucleotide compounds
CA3235262A1 (en) 2021-10-22 2023-04-27 Amgen Inc. Rnai constructs for inhibiting gpam expression and methods of use thereof
CA3235963A1 (en) * 2021-11-15 2023-05-19 Novartis Ag Branched triple lipid-modified nucleic acid compounds
JP7877458B2 (ja) 2021-12-01 2026-06-22 ディセルナ ファーマシューティカルズ インコーポレイテッド Ａｐｏｃ３発現を調節するための組成物及び方法
MX2024010419A (es) 2022-02-25 2024-09-05 Amgen Inc Metodos de preparacion de sustancias farmacologicas liquidas de alta concentracion.
CA3262515A1 (en) 2022-07-25 2024-02-01 Amgen Inc. ARNI CONSTRUCTIONS AND METHODS FOR INHIBITING FAM13A EXPRESSION
KR20250119645A (ko) 2022-12-16 2025-08-07 암젠 인크 Ttr 발현을 억제하기 위한 rnai 구성체 및 이의 사용 방법
KR20260089666A (ko) * 2023-09-14 2026-06-15 아이오니스 파마수티컬즈, 인코포레이티드 Apociii 발현을 감소시키기 위한 화합물 및 방법
WO2025252196A1 (zh) * 2024-06-06 2025-12-11 苏州吉玛基因股份有限公司 一种用于抑制补体成分C5表达的siRNA及其应用和产品
WO2025259726A1 (en) 2024-06-11 2025-12-18 Amgen Inc. Methods for separating diastereomers of double-stranded oligonucleotides
WO2025260042A1 (en) 2024-06-14 2025-12-18 Amgen Inc. Rnai constructs and methods for inhibiting cnr1 expression
WO2026006275A2 (en) 2024-06-26 2026-01-02 Amgen Inc. Rnai constructs and methods for inhibiting expression of inhbe

Citations (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2010083615A1 (en) * 2009-01-26 2010-07-29 Protiva Biotherapeutics, Inc. Compositions and methods for silencing apolipoprotein c-iii expression
WO2012177947A2 (en) * 2011-06-21 2012-12-27 Alnylam Pharmaceuticals, Inc. Compositions and methods for inhibition of expression of apolipoprotein c-iii (apoc3) genes
WO2014127268A2 (en) * 2013-02-14 2014-08-21 Isis Pharmaceuticals, Inc. Modulation of apolipoprotein c-iii (apociii) expression in lipoprotein lipase deficient (lpld) populations

Family Cites Families (68)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US23861A (en) * 1859-05-03 Improvement in revolving fire-arms
US2699808A (en) 1944-10-06 1955-01-18 Mark W Lowe Apparatus for peeling tomatoes
US2699508A (en) 1951-12-21 1955-01-11 Selectronics Inc Method of mounting and construction of mounting for low frequency piezoelectric crystals
US3687808A (en) 1969-08-14 1972-08-29 Univ Leland Stanford Junior Synthetic polynucleotides
US5367066A (en) 1984-10-16 1994-11-22 Chiron Corporation Oligonucleotides with selectably cleavable and/or abasic sites
FR2575751B1 (fr) 1985-01-08 1987-04-03 Pasteur Institut Nouveaux nucleosides de derives de l'adenosine, leur preparation et leurs applications biologiques
EP0366685B1 (en) 1987-06-24 1994-10-19 Howard Florey Institute Of Experimental Physiology And Medicine Nucleoside derivatives
US5175273A (en) 1988-07-01 1992-12-29 Genentech, Inc. Nucleic acid intercalating agents
US5134066A (en) 1989-08-29 1992-07-28 Monsanto Company Improved probes using nucleosides containing 3-dezauracil analogs
US5130302A (en) 1989-12-20 1992-07-14 Boron Bilogicals, Inc. Boronated nucleoside, nucleotide and oligonucleotide compounds, compositions and methods for using same
US5459255A (en) 1990-01-11 1995-10-17 Isis Pharmaceuticals, Inc. N-2 substituted purines
US5681941A (en) 1990-01-11 1997-10-28 Isis Pharmaceuticals, Inc. Substituted purines and oligonucleotide cross-linking
US5587470A (en) 1990-01-11 1996-12-24 Isis Pharmaceuticals, Inc. 3-deazapurines
JPH0874B2 (ja) 1990-07-27 1996-01-10 アイシス・ファーマシューティカルス・インコーポレーテッド 遺伝子発現を検出および変調するヌクレアーゼ耐性、ピリミジン修飾オリゴヌクレオチド
US5432272A (en) 1990-10-09 1995-07-11 Benner; Steven A. Method for incorporating into a DNA or RNA oligonucleotide using nucleotides bearing heterocyclic bases
US5594121A (en) 1991-11-07 1997-01-14 Gilead Sciences, Inc. Enhanced triple-helix and double-helix formation with oligomers containing modified purines
TW393513B (en) 1991-11-26 2000-06-11 Isis Pharmaceuticals Inc Enhanced triple-helix and double-helix formation with oligomers containing modified pyrimidines
US5484908A (en) 1991-11-26 1996-01-16 Gilead Sciences, Inc. Oligonucleotides containing 5-propynyl pyrimidines
EP0637965B1 (en) 1991-11-26 2002-10-16 Isis Pharmaceuticals, Inc. Enhanced triple-helix and double-helix formation with oligomers containing modified pyrimidines
WO1994014226A1 (en) 1992-12-14 1994-06-23 Honeywell Inc. Motor system with individually controlled redundant windings
ES2128535T3 (es) 1993-05-12 1999-05-16 Novartis Ag Nucleosidos y oligonucleotidos con grupos 2'-eter.
US5502177A (en) 1993-09-17 1996-03-26 Gilead Sciences, Inc. Pyrimidine derivatives for labeled binding partners
US5457187A (en) 1993-12-08 1995-10-10 Board Of Regents University Of Nebraska Oligonucleotides containing 5-fluorouracil
US5596091A (en) 1994-03-18 1997-01-21 The Regents Of The University Of California Antisense oligonucleotides comprising 5-aminoalkyl pyrimidine nucleotides
US5525711A (en) 1994-05-18 1996-06-11 The United States Of America As Represented By The Secretary Of The Department Of Health And Human Services Pteridine nucleotide analogs as fluorescent DNA probes
TW520293B (en) 1995-11-22 2003-02-11 Univ Johns Hopkins Med Delivery system to enhance cellular uptake of biomolecules
US5656408A (en) 1996-04-29 1997-08-12 Xerox Corporation Coated carrier particles
US6770748B2 (en) 1997-03-07 2004-08-03 Takeshi Imanishi Bicyclonucleoside and oligonucleotide analogue
JP3756313B2 (ja) 1997-03-07 2006-03-15 武 今西 新規ビシクロヌクレオシド及びオリゴヌクレオチド類縁体
US6794499B2 (en) 1997-09-12 2004-09-21 Exiqon A/S Oligonucleotide analogues
US6300319B1 (en) 1998-06-16 2001-10-09 Isis Pharmaceuticals, Inc. Targeted oligonucleotide conjugates
ATE356824T1 (de) 1999-05-04 2007-04-15 Santaris Pharma As L-ribo-lna analoge
US6525191B1 (en) 1999-05-11 2003-02-25 Kanda S. Ramasamy Conformationally constrained L-nucleosides
US7491805B2 (en) 2001-05-18 2009-02-17 Sirna Therapeutics, Inc. Conjugates and compositions for cellular delivery
CN100406065C (zh) 2000-12-01 2008-07-30 细胞工厂治疗公司 糖基化/半乳糖基化肽、双官能接头和核苷酸单体/多聚体的缀合物以及相关的组合物和使用方法
CA2504694C (en) 2002-11-05 2013-10-01 Isis Pharmaceuticals, Inc. Polycyclic sugar surrogate-containing oligomeric compounds and compositions for use in gene modulation
US8604183B2 (en) 2002-11-05 2013-12-10 Isis Pharmaceuticals, Inc. Compositions comprising alternating 2′-modified nucleosides for use in gene modulation
US7598227B2 (en) 2003-04-16 2009-10-06 Isis Pharmaceuticals Inc. Modulation of apolipoprotein C-III expression
US7723509B2 (en) 2003-04-17 2010-05-25 Alnylam Pharmaceuticals IRNA agents with biocleavable tethers
WO2004106356A1 (en) 2003-05-27 2004-12-09 Syddansk Universitet Functionalized nucleotide derivatives
DK1661905T3 (da) 2003-08-28 2012-07-23 Takeshi Imanishi Hidtil ukendte syntetiske nukleinsyrer af N-O-krydsbindingstype
WO2005027962A1 (en) 2003-09-18 2005-03-31 Isis Pharmaceuticals, Inc. 4’-thionucleosides and oligomeric compounds
WO2006031461A2 (en) 2004-09-09 2006-03-23 Isis Pharmaceuticals, Inc. Pyrrolidinyl groups for attaching conjugates to oligomeric compounds
US20060148740A1 (en) 2005-01-05 2006-07-06 Prosensa B.V. Mannose-6-phosphate receptor mediated gene transfer into muscle cells
US7569686B1 (en) 2006-01-27 2009-08-04 Isis Pharmaceuticals, Inc. Compounds and methods for synthesis of bicyclic nucleic acid analogs
DE602007009487D1 (de) 2006-01-27 2010-11-11 Isis Pharmaceutical Inc 6-modifizierte bicyclische nukleinsäureanaloga
US7666854B2 (en) 2006-05-11 2010-02-23 Isis Pharmaceuticals, Inc. Bis-modified bicyclic nucleic acid analogs
CA2651453C (en) 2006-05-11 2014-10-14 Isis Pharmaceuticals, Inc. 5'-modified bicyclic nucleic acid analogs
US20100190837A1 (en) 2007-02-15 2010-07-29 Isis Pharmaceuticals, Inc. 5'-Substituted-2-F' Modified Nucleosides and Oligomeric Compounds Prepared Therefrom
DK2170917T3 (da) 2007-05-30 2012-10-08 Isis Pharmaceuticals Inc N-Substituerede bicycliske nukleinsyreanaloge med aminomethylenbro
WO2008154401A2 (en) 2007-06-08 2008-12-18 Isis Pharmaceuticals, Inc. Carbocyclic bicyclic nucleic acid analogs
AU2008272918B2 (en) 2007-07-05 2012-09-13 Isis Pharmaceuticals, Inc. 6-disubstituted bicyclic nucleic acid analogs
EP2229186A2 (en) 2007-12-04 2010-09-22 Alnylam Pharmaceuticals Inc. Carbohydrate conjugates as delivery agents for oligonucleotides
US8575123B2 (en) 2008-04-11 2013-11-05 Tekmira Pharmaceuticals Corporation Site-specific delivery of nucleic acids by combining targeting ligands with endosomolytic components
WO2009143369A2 (en) 2008-05-22 2009-11-26 Isis Pharmaceuticals, Inc. Method of preparing nucleosides and analogs thereof without using chromatography
WO2010036698A1 (en) 2008-09-24 2010-04-01 Isis Pharmaceuticals, Inc. Substituted alpha-l-bicyclic nucleosides
EP2358397B1 (en) 2008-10-24 2020-01-01 Ionis Pharmaceuticals, Inc. 5' and 2' bis-substituted nucleosides and oligomeric compounds prepared therefrom
EP2447274B1 (en) 2008-10-24 2017-10-04 Ionis Pharmaceuticals, Inc. Oligomeric compounds and methods
AT507215B1 (de) 2009-01-14 2010-03-15 Boehler Edelstahl Gmbh & Co Kg Verschleissbeständiger werkstoff
WO2011115818A1 (en) 2010-03-17 2011-09-22 Isis Pharmaceuticals, Inc. 5'-substituted bicyclic nucleosides and oligomeric compounds prepared therefrom
WO2011139702A2 (en) * 2010-04-28 2011-11-10 Isis Pharmaceuticals, Inc. Modified nucleosides and oligomeric compounds prepared therefrom
US9290760B2 (en) 2010-09-15 2016-03-22 Alnylam Pharmaceuticals, Inc. Modified iRNA agents
US8603994B2 (en) * 2010-11-11 2013-12-10 Valted, Llc Transcriptional repression leading to Parkinson's disease
SG194671A1 (en) * 2011-04-27 2013-12-30 Isis Pharmaceuticals Inc Modulation of apolipoprotein ciii (apociii) expression
US10023861B2 (en) * 2011-08-29 2018-07-17 Ionis Pharmaceuticals, Inc. Oligomer-conjugate complexes and their use
EP2751269B1 (en) * 2011-08-29 2016-03-23 Ionis Pharmaceuticals, Inc. Methods and compounds useful in conditions related to repeat expansion
US9984408B1 (en) 2012-05-30 2018-05-29 Amazon Technologies, Inc. Method, medium, and system for live video cooperative shopping
US9778708B1 (en) 2016-07-18 2017-10-03 Lenovo Enterprise Solutions (Singapore) Pte. Ltd. Dual sided latching retainer for computer modules

Patent Citations (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2010083615A1 (en) * 2009-01-26 2010-07-29 Protiva Biotherapeutics, Inc. Compositions and methods for silencing apolipoprotein c-iii expression
WO2012177947A2 (en) * 2011-06-21 2012-12-27 Alnylam Pharmaceuticals, Inc. Compositions and methods for inhibition of expression of apolipoprotein c-iii (apoc3) genes
WO2014127268A2 (en) * 2013-02-14 2014-08-21 Isis Pharmaceuticals, Inc. Modulation of apolipoprotein c-iii (apociii) expression in lipoprotein lipase deficient (lpld) populations

Non-Patent Citations (1)

* Cited by examiner, † Cited by third party
Title
WALT F. LIMA ET AL, "Single-Stranded siRNAs Activate RNAi in Animals", CELL, 2012, vol. 150, no. 5, pages 883 - 894, doi:10.1016/j.cell.2012.08.014, ISSN 0092-8674 *

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